ADVL0319: A Phase I Study of CC-5013 in Pediatric Patients with Relapsed / Refractory Solid Tumors or Myelodysplastic Syndrome
What is the purpose of this study?
The goals of this study are:
- to find the highest dose of CC-5013 that can be given without causing severe side effects
- to learn what kind of side effects CC-5013 can cause
- to learn more about the pharmacology (how the body handles the drug) of CC-5013
- to determine whether CC-5013 is a beneficial treatment for the tumor
- to see if children with a kind of bone marrow disease called myleodysplatic syndrome (MDS) get any benefit from CC-5013
Who will be included in this study?
Patients who are greater than 12 months old or less than or equal to 21 years old with relapsed or refractory solid tumors or myelodysplastic syndrome may be eligible to participate in this clinical trial.
What is involved?
Patients may be in the study for up to 12 courses of therapy (about 1 year) if they are responding to therapy. After patients are finished taking CC-5013, they will be asked to visit their doctor's office for follow-up exams. We would like to keep track of each patient's medical condition for as long as we are able.
Before you begin the study
Patients will need to have the following exams, tests, or procedures to find out if they can be in the study. These exams, tests, or procedures are part of regular cancer care and may be done even if patient do not join the study. If the tests have been performed recently, they may not need to be repeated. This will be up to the research doctor.
- A medical history
- Physical exam
- Vital signs (blood pressure, pulse, temperature)
- Blood tests
- Urine tests
- Pregnancy test (for women who could have children)
- Standard x-rays, CT scans, MRI scans, or other tests as needed to check the tumor
- Bone marrow exam (MDS patients only)
During the study
Patients will also be given the following tests and procedures during the study. They are part of regular cancer care, but may be done more often for patients participating in this study.
- Physical exam
- Blood tests
- Pregnancy test (for women who could have children)
- Scans to evaluate the tumor
- Bone marrow exam (MDS patients only)
- Other optional laboratory studies
What are the benefits?
There may be no direct benefit to patients who agree to take part in this research study. It is unlikely that patients will experience significant benefit or cure from this drug, CC-5013. The potential benefit is that CC-5013 may cause the cancer to stop growing or go into partial remission for a period of time or it may lessen symptoms. It may lessen the symptoms, such as pain, that are caused by the cancer. Because there is not much information about the effects of CC-5013 in humans, we do not know if patients will benefit from taking part in this study. The knowledge gained from this study may benefit others. The disease may continue to become worse despite taking this drug.
Clinical studies are essential in finding new and improved treatments and diagnostic tools.
Will I get all the facts about the study?
Parents interested in having their child participate will be scheduled with a physician who will thoroughly explain all of the details of the study. Your physician will review the consent form with you and will be sure that all of your questions are answered. The consent form goes over all of the procedures, the risks, the benefits, the compensation, who to contact with questions or concerns and more. Study procedures will not begin until a parent/guardian has signed this form and, if of age, the child has given at minimum his/her verbal agreement.
What are the risks?
A detailed list of side effects will be provided to those patients interested in knowing more about the study.
Who should I contact for more information?
Peggy Kaiser, RN, BSN
Cincinnati Children's Hospital Medical Center
Division of Hematology / Oncology
3333 Burnet Ave., Cincinnati, OH 45229-3039
Phone: 513-636-2799
cancer@cchmc.org
